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‘Please don’t let us die’: Three cystic fibrosis sufferers beg the NHS to fund ‘wonder drug’

It’s been hailed as a wonder drug with the potential to extend the lives of thousands of people with cystic fibrosis.   

But patients across the UK still can’t get their hands on Orkambi – despite it being a year since Theresa May promised to take action.

The drug, which slows lung deterioration, received its European licence three-and-a-half years ago, in which time over 200 people have died from the cruel condition. 

However, the National Institute of Health and Care Excellence (NICE) has been unable to reach an agreement with its manufacturer.

The Goverment-run body and NHS England have been in a deadlock with US firm Vertex over the drug, which costs an estimated £104,000 per patient per year. 

On the anniversary of the Prime Minister’s pledge on May 16, furious campaigners marched to 10 Downing Street to share their frustrations.

Mrs May responded last night by telling the firms to do ‘everything they can’ to break the deadlock. 

And MailOnline has spoken to three cystic fibrosis sufferers, who have begged the parties to make headway.

Hannah Chew née Lindley, 23, Shiloh Howells, nine, and Lorcán Maguire, two, are desperate for the chance to try Orkambi. 

Three cystic fibrosis sufferers have begged the NHS to fund a ‘wonder drug’ to combat the crippling condition. Lorcán Maguire, of Downpatrick, Northern Ireland, is two

Shiloh Howells, nine, has lungs so fragile that she is expected to have a risky double lung transplant by the age of 12. Her mother, Nichola Howells, said it's heartbreaking knowing Orkambi could prevent the operation

Shiloh Howells, nine, has lungs so fragile that she is expected to have a risky double lung transplant by the age of 12. Her mother, Nichola Howells, said it’s heartbreaking knowing Orkambi could prevent the operation 

Hannah Chew née Lindley, 23, of Leeds, said CF has stolen her joy, childhood and her best friend – she tragically had to watch her sister, Iona Kesteven, pass away from the disease. Pictured on her wedding day in August 2018

Hannah Chew née Lindley, 23, of Leeds, said CF has stolen her joy, childhood and her best friend – she tragically had to watch her sister, Iona Kesteven, pass away from the disease. Pictured on her wedding day in August 2018

WHAT IS ORKAMBI AND WHO WOULD BENEFIT?

Orkambi can slow decline in lung function – the main cause of death among people with cystic fibrosis – by 42 per cent.  

CF is a progressive, genetic disease that causes the mucus in various organs to become thick and sticky and trap bacteria.

It causes persistent lung infections and limits the ability to breathe over time, limiting the life expectancy to mid-40s.

There are almost 3,000 people in Great Britain who have two copies of the F508del mutation – around half of patients – that could benefit from Orkambi, according to the 2014 UK Cystic Fibrosis Registry Report. 

There are 2,834 people in England, 243 people in Scotland, 118 people in Wales and 101 people in Northern Ireland.

Vertex refused an offer from NHS England of £500million for a five-year supply, the biggest offer it has ever made, and talks have completely broken down.

The drug is only ever prescribed on rare compassionate grounds, which many charities say is too late.  

Mrs May said she wanted a ‘speedy resolution’ in the ongoing negotiations so CF patients could have access to Orkambi and Symkevi –  another drug that treats the root cause of the condition but is also not available on the NHS.

Since then, there have been no resolutions.

Vertex reached an agreement with Scotland for access to Orkambi and Symkevi in December, but the fight for the drug is still raging on in England, Wales and Northern Ireland. 

Yesterday, the Cystic Fibrosis Trust handed a letter to Theresa May at number 10 Downing Street.

Zoey Jones, the distressed mother of one-year-old Eve, who has cystic fibrosis, was also there to give 65 roses – a term used by children to help them pronounce the name of the condition. 

David Ramsden, chief executive of the Cystic Fibrosis Trust, said: ‘This tragic situation cannot continue. Every day the health of thousands of people in this country is deteriorating – and this is damage to their lungs that can’t be undone.’

‘We’re asking for Prime Minister, Theresa May to attend the Westminster Hall debate on Monday 10 June and update the house on what the next steps are.’     

‘My sister died waiting for that drug’

Hannah Chew née Lindley, 23, said CF has stolen her joy, childhood and her best friend – she tragically had to watch her sister, Iona Kesteven, pass away from the condition.

Hannah Chew née Lindley, 23 said her and her sister Iona, right, who passed away, are best friends

Hannah Chew née Lindley, 23 said her and her sister Iona, right, who passed away, are best friends

When Hannah looks back on her childhood, she mostly remembers Iona, who died at the age of 19, being ill. Pictured on holiday

When Hannah looks back on her childhood, she mostly remembers Iona, who died at the age of 19, being ill. Pictured on holiday

Around the age of eight, Hannah remembers the illness taking a tighter hold of Iona, and they were put in separate bedrooms. Pictured, in hospital, date is unclear

Around the age of eight, Hannah remembers the illness taking a tighter hold of Iona, and they were put in separate bedrooms. Pictured, in hospital, date is unclear

Hannah, of Leeds, who is finishing exams to become a music teacher, isn’t afraid to admit that her life with CF, apart from some happy memories, has been ‘horrific’.

She said: ‘A lot of people mask it over which annoys me because it needs to take it seriously.

‘It’s stolen my joy, health and mobility. It stole my childhood and my best friend – my sister. I had to watch her die.

‘There are some days you feel better and there are others you question why you are on this planet.’

When Hannah looks back on her childhood, she mostly remembers Iona, who died at the age of 19, being ill.

She said: ‘We lived life permanently in a touch and go situation. I remember my sister being taken ill all the time and not knowing if she was going to live or die and constantly living with the fear of – is this the time she dies?’

In November 2015, Iona's lung collapsed due to multiple lung infections and she was rushed back into hospital. A week later she passed away. Pictured on holiday before

In November 2015, Iona’s lung collapsed due to multiple lung infections and she was rushed back into hospital. A week later she passed away. Pictured on holiday before

Hannah’s parents tried to make their lives as normal as possible, taking the girls to ballet, piano lessons and performing groups.

She said: ‘We even went to the theatre to perform from the hospital. We did our makeup in the hospital. We didn’t want to be another number of kids stuck on the ward. We minimalised being in there as much as possible.’

But around the age of eight, Hannah remembers the illness taking a tighter hold of Iona, and they were put in separate bedrooms.

People with CF grow bugs in their lungs which are usually harmless to people who don’t have the condition, but can be easily transmitted from one person with CF to another, including siblings, and be very harmful. 

Hannah said: ‘I felt a lot of guilt because I wasn’t as poorly as she was.’

Hannah, who married Nathan Chew, 23 (pictured), in August 2018, isn't afraid to admit that her life with CF, apart from some happy memories, has been 'horrific'

Hannah, who married Nathan Chew, 23 (pictured), in August 2018, isn’t afraid to admit that her life with CF, apart from some happy memories, has been ‘horrific’

Iona was known to her consultants as being a complex patient, but Hannah believes it was May 2015, when Iona picked up a stomach bug on the flight home from a holiday, that things went severely downhill.

In November 2015, her lung collapsed due to multiple lung infections and she was rushed back into hospital. A week later she passed away.

Hannah, who married Nathan Chew, 23, in August 2018, said: ‘One of the consultants was in shock because they believed she would have survived longer. 

‘She could still be here with Orkambi. My sister died waiting for that drug. It was approved on compassionate grounds two weeks after her death but it was too late.

‘The healthier you are, the better chance you have, so they shouldn’t be waiting for them to get to a certain point.’

Hannah is also frustrated that CF patients with other mutations are given drugs that can treat them – despite being happy for them.

She said: ‘It’s disgusting. It’s like saying we will treat breast cancer but not lung cancer. Or type 1 diabetes but not type 2.’ 

‘Orkambi could help me avoid a double lung transplant at the age of 12’

Shiloh Howells, nine, has lungs so fragile that she is expected to have a risky double lung transplant by the age of 12.

Doctors say the extremity of her condition is far worse than other CF patients her age, having had a lobectomy – surgery to remove part of her damaged right lung, in October 2017.

Her mother, Nichola Howells, 40, said just knowing Orkambi exists is heartbreaking, considering it could preserve what lung function Shiloh has left.

Shiloh, pictured with her mother, Ms Howells, and grandmother, has very fragile lungs with a  baseline lung function of around 60 per cent

Shiloh, pictured with her mother, Ms Howells, and grandmother, has very fragile lungs with a  baseline lung function of around 60 per cent

Shiloh's lungs so fragile that she is expected to have a risky double lung transplant by the age of 12. Pictured in hospital where she goes for routine treatment every two months

Shiloh’s lungs so fragile that she is expected to have a risky double lung transplant by the age of 12. Pictured in hospital where she goes for routine treatment every two months

Ms Howells said: ‘It would mean everything to get Orkambi within that time. With a lung transplant, there is no guarantee you’ll survive it. It’s still life-threatening.

‘Her condition isn’t stable and it’s frightening. If she had that drug, within a year who knows what an improvement it wold make.

‘It’s soul destroying for us. It’s money and finance over a life. We are angry and frustrated.’

In March 2018, ex-Health Secretary Jeremy Hunt visited Great Ormond Street Hospital in London and was welcomed by Shiloh who gave him a teddy bear, the hospital’s mascot.

But, according to Ms Howells, as soon as he was told Shiloh was being treated for CF, he swiftly moved on amid the price-war outrage.

Ms Howells said: ‘While she was in mid conversation he stood up and walked away. Maybe he was worried we would bombard him with questions. We were all taken back.’

From the age of six weeks, Shiloh has been having routine hospital admissions every three months to receive treatment to avoid infections. Pictured, with medication

From the age of six weeks, Shiloh has been having routine hospital admissions every three months to receive treatment to avoid infections. Pictured, with medication

In March 2018, ex Health Secretary Jeremy Hunt visited Great Ormond Street Hospital and was welcomed by Shiloh. But Ms Howells claims he was dismissive as soon as he heard she had CF

In March 2018, ex Health Secretary Jeremy Hunt visited Great Ormond Street Hospital and was welcomed by Shiloh. But Ms Howells claims he was dismissive as soon as he heard she had CF

From the age of six weeks, Shiloh has been having routine hospital admissions every three months to receive treatment to avoid infections.

WHAT IS CYSTIC FIBROSIS? 

Cystic fibrosis is an incurable genetic disease that affects around 70,000 people worldwide.

A defective gene causes a build-up of mucus in the airways, making it increasingly difficult to breathe over time.

Mucus also blocks the natural release of digestive enzymes, meaning the body does not break down food as it should.

Signs and symptoms:

  • A persistent cough that produces thick mucus (sputum)
  • Wheezing
  • Breathlessness
  • Exercise intolerance
  • Repeated lung infections
  • Inflamed nasal passages or a stuffy nose 

While healthy people cough naturally, that does not happen for people with CF.

Eventually, lung function depletes to the point that sufferers will need a double lung transplant to survive.   

Source: Cystic Fibrosis Foundation 

‘But she wasn’t making the three month mark before getting ill,’ Mrs Howell said. ‘So she now goes to Great Ormond Street Hospital every two months for antibiotic and IV treatment, staying between two weeks and three months.’

During Shiloh’s longest stay, aged eight, she stayed for three months when her lung function dropped to 36 per cent.

Her baseline lung function is 60 per cent, but it used to sit around the 75 per cent mark.

However, always positive and smiling, Shiloh managed to go to school for a solid two months recently – the longest period in two years.

Ms Howells, who has an older daughter called Essence, 20, said: ‘She’s been on the brink but thankfully she picks herself up. But it’s getting harder and harder.

‘Her lungs are already very delicate. To prevent other stories like hers, patients should be given Orkambi as early as possible.

‘CF is all about prevention. We are forever throwing medication at the sufferers to try and make them as comfortable as we can. But Orkambi tackles the genetic side.’

‘It’s a lot for a wee man, and he is still going to die’

Having just turned two, Lorcán Maguire, of Downpatrick, Northern Ireland, is still unaware of how different his life is to other children’s.

Every day he has 18 tablets, two nebuliser treatment for antibiotics, another nebuliser for a drug to thin the mucus, two fifteen minutes of physiotherapy and vitamins because he can’t absorb them properly from food.

His mother, Jen Maguire, 34, said: ‘It’s a lot for a wee man, and he is still going to die. 

‘The estimate now is mid 40s. But Orkambi has shown to add 20 years to current life expectancy. 

‘It’s such a terrible situation. I wish he had the same life expectancy as his sister.

Jen Maguire, 34, said her family, including husband Dermot, 45, 11-month-daughter, Saorla (left), and son Lorcán, who has CF, are on the verge of moving to the Republic of Ireland to get Orkambi

Jen Maguire, 34, said her family, including husband Dermot, 45, 11-month-daughter, Saorla (left), and son Lorcán, who has CF, are on the verge of moving to the Republic of Ireland to get Orkambi 

‘He just thinks his life is like everyone else’s. I hope we get Orkambi before he is aware of the situation.

‘It must be so harder for older people with CF who know the government just don’t care.’

Mrs Maguire, who also has an 11-month-daughter, Saorla, said the condition has ruined Lorcán’s infancy – and it’s only the beginning.

She said: ‘He can play with other children but we are constantly using bacteria gel and keeping eyes opening and ears open. 

‘As soon as you hear a cough or smell a cigarette we would whip him away immediately.’

When Lorcán was diagnosed with CF two weeks after birth, Mrs Maguire and her husband, Dermot Maguire, 45, a social worker, had barely heard of the condition which would change the whole family’s life.

They are on the verge of moving to the Republic of Ireland, where Orkambi has been given to eligible patients since 2017.

Mrs Maguire said the condition has ruined Lorcán's infancy – and it's only the beginning. Currently he is trying to fight an infection called pseudomonas aeruginosa, which has been persistently difficult to treat since November 2018. Pictured, the two together while sorting out one of Lorcán's medications

Mrs Maguire said the condition has ruined Lorcán’s infancy – and it’s only the beginning. Currently he is trying to fight an infection called pseudomonas aeruginosa, which has been persistently difficult to treat since November 2018. Pictured, the two together while sorting out one of Lorcán’s medications

Mrs Maguire said she wishes Lorcán had the same life expectancy as his sister, pictured

Mrs Maguire said she wishes Lorcán had the same life expectancy as his sister, pictured

Since January, Orkambi has been licenced – but not funded – for two-year-olds in the UK.

WHAT DID THERESA MAY SAY? 

On the 16th May, Theresa May told MPs in the House of Commons that she had sought a ‘speedy resolution’ to the price row for Orkambi.

She said: ‘Cystic fibrosis is obviously a terrible, life-limiting condition, and it is right that patients should have access to cost-effective, innovative medicines and technologies. 

‘The issue has been taken up by Members from across the House and, as the hon. Lady mentioned, there is an ongoing dialogue between NHS England and Vertex, but I am keen to see a speedy resolution to the negotiations. I understand that several Members have asked to see me about the issue, and I am happy for that to happen.’

Mrs Maguire, who has put her PhD studies in education on hold because she is too stressed to study, said: ‘If nothing has happened by summer holidays we are thinking of renting out our house and moving down there.

‘We are hoping something will happen before then, because it’s not ideal. It’s scary knowing how quickly things worsen with CF.’

Currently Lorcán is trying to fight an infection called pseudomonas aeruginosa, which has been persistently difficult to treat since November 2018.

He’s just returned from a ten day stint in hospital, isolated in a room to avoid contracting more infections from other CF patients.

Drugs were being pumped through an IV line, after three months of antibiotics failed to treat the infection. The doctors are still unsure if it has gone.

Mrs Maguire said: ‘Can you imagine a two-year-old confined to a room? He was screaming and screaming as they put the IV lines in and he shakes every time.

‘It’s worrying because the infection is becoming more resistant to drugs but it causes a lot of damage.

‘Orkambi has been shown to reduce bacteria and hospitalisation by 60 per cent. It deals with the root cause by making the mucus less sticky.

‘It’s not good to pump children with these drugs that only treat symptoms when you could stop something in the first place.’

MailOnline contacted NICE and Vertex for comment, who both said talks are ongoing. 

In a statement, Vertex said: ‘Vertex is in ongoing discussions with NHS England and NICE. We are unable to comment further at this time .’

NICE said: ‘We would welcome re-engagement from Vertex. Vertex, NICE and NHS England met in March and have agreed to continue discussions.’  

WHAT IS ORKAMBI?

Orkambi is a medication licensed for use by people in the UK with cystic fibrosis, but it is not offered on the NHS except in extreme circumstances.

The drug targets the F508del gene mutation, which affects about 50 per cent of all people with cystic fibrosis.

It is made of a combination of drugs – lumacaftor and ivacaftor – which work together to keep a healthy balance of salt and water in the lungs.  

There may be more than 3,000 people with life-limiting cystic fibrosis in the UK who could benefit from the drug.

But the National Institute for Health and Care Excellence (Nice) said in 2016  may not be cost-effective enough, at £104,000 per patient per year, to be offered on the NHS.

More than 117,000 people have signed a petition calling on the Government to make Orkambi available on the NHS, and Parliament discussed the petition in March this year.

Nice is expected to review its stance on the drug in July 2019. 

Daniel Bodio, an engineer from Swindon in his mid-30s, was prescribed the drug on compassionate grounds.

He said it improved his lung function from 25 per cent – when he was facing having to have a transplant – to 39 per cent within three weeks.  

He told MailOnline last year: ‘The drug should be available to everyone – it brings normality to what is otherwise a difficult existence.’ 

Read more at DailyMail.co.uk


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